Pfizer’s phase 3 Duchenne muscular dystrophy (DMD) gene therapy failure has blown a $230 million hole in the New York pharma's second quarter financials (PDF). The drugmaker disclosed the discontinuation of the candidate—and the financial fallout—alongside the axing of a respiratory syncytial virus (RSV) combination vaccine.

辉瑞的3期杜兴肌营养不良症（DID）基因治疗失败，导致这家纽约制药公司第二季度财务出现2.3亿美元的漏洞（PDF）。该制药商披露了该候选疫苗的停产以及财务影响，同时还取消了呼吸道合胞病毒（RSV）联合疫苗。

The Big Pharma reported the failure of a phase 3 clinical trial for the DMD gene therapy fordadistrogene movaparvovec in June. At that time, Pfizer was still evaluating the next steps for the program. Six weeks later, the company has confirmed there will be no significant next steps, jettisoning the candidate from its pipeline and taking a $230 million charge in the process.

大型制药公司于6月报告称，DMZ基因疗法fordadistrogene movaparvovec的3期临床试验失败。当时，辉瑞仍在评估该计划的下一步步骤。六周后，该公司确认不会有重大的下一步行动，将该候选人从其管道中抛弃，并在此过程中收取2.3亿美元的费用。

Pfizer’s decision brings a troubled program to an end. One month before the phase 3 fail, Pfizer paused dosing in the crossover portion of the phase 3 trial after a young boy in another study of the candidate died. The company also laid off 150 workers in North Carolina due to the gene therapy's failure.

辉瑞的决定结束了一个陷入困境的项目。在第三阶段试验失败前一个月，在另一项候选人研究中的一名小男孩死亡后，辉瑞暂停了第三阶段试验的交叉部分给药。由于基因疗法失败，该公司还在北卡罗来纳州解雇了150名员工。

Sarepta Therapeutics’ DMD gene therapy Elevidys has also suffered setbacks, notably when it failed to hit the primary goal of a pivotal study, but the biotech has continued to grow sales and push to reach more patients. The FDA expanded Elevidys’ label in June.

Sarepta Therapeutics的DMZ基因疗法Elevidys也遭遇了挫折，特别是它未能实现关键研究的主要目标，但该生物技术继续增长销售额并推动接触更多患者。FDA于六月扩大了Elevidys的标签范围。

UPDATE: Pfizer's phase 3 gene therapy trial fails to improve function for boys with Duchenne muscular dystrophy

最新消息：辉瑞的三期基因治疗试验未能改善杜兴肌营养不良症男孩的功能

Pfizer disclosed the discontinuation of the DMD gene therapy alongside news that it is stopping work on a phase 2 respiratory vaccine combination. The candidate, PF-07960613, combined vaccines designed to protect against RSV and COVID-19.

辉瑞公司透露，该公司已停止DMZ基因疗法，并有消息称该公司将停止2期呼吸道疫苗组合的研发。候选疫苗PF-07960613是旨在预防RSV和COVID-19的组合疫苗

No candidate by that name is listed on ClinicalTrials.gov but the database does include a Pfizer phase 2 trial of a combined vaccine for RSV and COVID-19. The study, which ran from June 2023 to the start of 2024, enrolled more than 1,000 people aged 65 years and older to receive vaccine regimens including a combination of RSVpreF and bivalent BNT162b2 on top of a quadrivalent influenza shot.

ClinicalTrials.gov上没有列出该名称的候选人，但该数据库确实包括辉瑞公司一项针对RSV和COVID-19联合疫苗的II期试验。这项研究从2023年6月持续到2024年初，招募了超过1，000名65岁及以上的人接受疫苗方案，包括在四价流感疫苗的基础上联合使用RSVpreF和二价BNT 162 b2。

RSVpreF is the RSV protein subunit vaccine that Pfizer sells as Abrysvo. Bivalent BNT162b2 is an updated version of Pfizer’s COVID-19 vaccine Comirnaty. The study assessed the rates of local reactions, systemic events and adverse events in people who received various combinations of the vaccines and placebo. Pfizer also looked at immune responses.

RSVpreF是辉瑞以Abrysvo名义销售的RSV蛋白亚单位疫苗。二价BNT 162 b2是辉瑞COVID-19疫苗Comirnaty的更新版本。该研究评估了接受各种疫苗和安慰剂组合的人的局部反应、全身事件和不良事件的发生率。辉瑞还研究了免疫反应。